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This drug just got the FDA's OK -- and an $89K price tag

14 February 2017

The U.S. Food and Drug Administration is now approved Emflaza (deflazacort), an old steroid treatment, long accessible outside the United States.

Essentially, Marathon is being rewarded for going through the process of getting a brand new drug approved, even though it has been on the market for years.

Critics say that Marathon is simply taking an old drug for a rare disease, and increasing the price for their own gain.

The first drug, eteplirsen, developed by Sarepta Therapeutics, gained FDA approval in September.

Here is the statement from the FDA.

The U.S. Food and Drug Administration last week approved a drug made by Marathon Pharmaceuticals to treat Duchenne muscular dystrophy (DMD), a devastating muscle-wasting disease that mainly affects young boys.

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Corticosteroids are commonly used to treat DMD across the world.

Deflazacort has always been used off-label for DMD treatment, but until now hadn't been approved to treat the condition. The drug is a common steroid used to treat Duchenne muscular dystrophy (DMD), a rare disorder that eats away at muscles and leaves many of its predominantly male victims dead by their late-20s. "We hope that this treatment option will benefit many patients with DMD".

The regulator gave a green light to Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with the rare genetic disorder, which causes progressive muscle deterioration and weakness in children, nearly always boys. DMD often appears in individuals with no family history of the condition. DMD affects about one of every 3,600 male infants worldwide according to U.S. News. As the condition progresses, chances of life-threatening conditions developing involving the respiratory system and the heart grow. Patients' life expectancy is usually 20 to 30 years. All of the study participants had documented dystrophin gene mutation and had experience muscular weakness before 5 years old. At week 12, patients in the Emflaza group had improvements in clinical assessment of muscle strength across a number of muscles vs patients taking placebo. An overall stability in average muscle strength was maintained through the end of study at week 52 in the deflazacort-treated patients. Patients in the Emflaza arm seemed to lose their walking ability later than patients in the placebo arm, though this was not statistically controlled for multiple comparisons.

The side effects caused by Emflaza are similar to those experienced with other corticosteroids. Other side effects include mood swings, elevated blood pressure, infection and serious skinj rashes. Patients receiving immunosuppressive doses of corticosteroids should not be given live or live attenuated vaccines.

The FDA also granted Emflaza priority review and fast track and orphan drug designations. "This also seems to be another example of gaming the Orphan Drug Act, which was meant to try and encourage research into new therapeutic entities for people who have rare diseases - and it doesn't seem like this is that".